Ayrmid Reports New Data on Motixafortide for Stem Cell Mobilization in Sickle Cell and Beta-Thalassemia

Motixafortide effectively mobilized stem cells, enabling access to gene therapies for patients.

Feb. 4, 2026 at 10:23am

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Ayrmid, the parent company of Gamida Cell, announced new real-world data on the use of motixafortide, a CXCR4 inhibitor, for mobilizing hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia. The results, presented at the TANDEM 2026 conference, showed that motixafortide effectively mobilized sufficient HSCs in 73% of patients, including those who had previously failed to collect enough cells with plerixafor. This enabled progress to gene therapy manufacturing, with 5 patients receiving their gene therapy and appropriately engrafting.

Why it matters

Collecting adequate stem cells remains a significant barrier to gene therapy for sickle cell disease, as many patients do not mobilize enough cells with standard approaches. These findings suggest that motixafortide may help overcome this challenge and expand access to potentially life-changing gene therapies for sickle cell disease and beta-thalassemia.

The details

Researchers from five treatment centers evaluated the real-world use of motixafortide in 15 patients aged 14-50. Each patient underwent one or more collection cycles over two or more days to obtain the number of stem cells required for gene therapy manufacturing. Motixafortide effectively mobilized 11 patients who had previously failed plerixafor, and 3 patients mobilized effectively with motixafortide used as first-line mobilization therapy. In 11 patients, sufficient cells were achieved to manufacture gene therapy for both sickle cell disease and beta-thalassemia. 5 patients have received their gene therapy and have appropriately engrafted, with manufacturing underway for 5 additional patients.

  • The results were presented at the 2025 Transplantation & Cellular Therapy meetings of ASTCT and CIBMTR (TANDEM), taking place from February 4-7, 2026 in Salt Lake City, UT.

The players

Ayrmid, Ltd.

The parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed to turn cells into powerful therapeutics.

Motixafortide

A long-acting CXCR4 inhibitor licensed by Ayrmid under the brand name APHEXDA, which is currently FDA approved in combination with filgrastim (G-CSF) for stem cell mobilization in multiple myeloma.

Dr. John Manis

Director Transfusion Medicine Service and Associate Professor of Pathology at Harvard Medical School.

Sickle Cell Disease

A severe inherited blood disorder caused by a genetic mutation that leads to misshapen, rigid red blood cells, which can obstruct blood flow and cause life-threatening complications.

Beta-Thalassemia

A genetic blood disorder characterized by reduced or absent production of beta-globin, a component of hemoglobin, resulting in anemia and associated symptoms.

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What they’re saying

“The approval of gene therapies for sickle cell disease has opened the door to transformative outcomes, but inadequate stem cell collection has significantly delayed manufacturing and prevented some patients from starting treatment. These findings suggest that motixafortide may help overcome this challenge, and with more studies, expand access to potentially life-changing therapies.”

— Dr. John Manis, Director Transfusion Medicine Service, Associate Professor of Pathology, Harvard Medical School (TANDEM 2026)

The takeaway

These findings demonstrate that motixafortide can effectively mobilize hematopoietic stem cells in patients with sickle cell disease and beta-thalassemia, enabling progress to gene therapy manufacturing and potentially expanding access to these transformative treatments for patients who have struggled to collect enough cells through standard approaches.